STAT+: Can ultrasound unlock the power of gene therapy? A startup makes big claims

For the last year, a small California startup has been making extraordinary claims about the ability of its technology to potentially treat Duchenne muscular dystrophy — and maybe a slew of other genetic diseases, too. The company, Sonothera, does not yet have clinical data. But it’s presented data in animals so stunning that other experts can’t quite wrap their minds around it. “I find it hard to believe,” said Eric Olson, molecular biology chair at UT-Southwestern Medical Center. “It seems a bit too good to be true,” said Jeffrey Chamberlain, a longtime Duchenne gene therapy expert at the University of Washington. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe